A differentiated β-globin gene replacement strategy uses heterologous introns to restore physiological expression.
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| Title: | A differentiated β-globin gene replacement strategy uses heterologous introns to restore physiological expression. |
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| Authors: | Wallace KA; Graphite Bio, Inc., South San Francisco, CA 94080, USA; Kamau Therapeutics, Inc., South San Francisco, CA 94080, USA., Gerstenberg TL; Graphite Bio, Inc., South San Francisco, CA 94080, USA., Ennis CL; Graphite Bio, Inc., South San Francisco, CA 94080, USA., Perez-Bermejo JA; Graphite Bio, Inc., South San Francisco, CA 94080, USA., Partridge JR; Graphite Bio, Inc., South San Francisco, CA 94080, USA., Bandoro C; Graphite Bio, Inc., South San Francisco, CA 94080, USA., Matern WM; Graphite Bio, Inc., South San Francisco, CA 94080, USA., Andreoletti G; Graphite Bio, Inc., South San Francisco, CA 94080, USA., Krassovsky K; Graphite Bio, Inc., South San Francisco, CA 94080, USA., Kabir S; Graphite Bio, Inc., South San Francisco, CA 94080, USA., Lalisan CD; Graphite Bio, Inc., South San Francisco, CA 94080, USA., Churi AR; Graphite Bio, Inc., South San Francisco, CA 94080, USA., Chew GM; Graphite Bio, Inc., South San Francisco, CA 94080, USA., Corbo L; Graphite Bio, Inc., South San Francisco, CA 94080, USA., Vincelette JE; Graphite Bio, Inc., South San Francisco, CA 94080, USA., Klasson TD; Graphite Bio, Inc., South San Francisco, CA 94080, USA., Silva BJ; Graphite Bio, Inc., South San Francisco, CA 94080, USA., Strukov YG; Graphite Bio, Inc., South San Francisco, CA 94080, USA., Quejarro BJ; Graphite Bio, Inc., South San Francisco, CA 94080, USA., Hill KA; Graphite Bio, Inc., South San Francisco, CA 94080, USA., Treusch S; Graphite Bio, Inc., South San Francisco, CA 94080, USA., Grogan JL; Graphite Bio, Inc., South San Francisco, CA 94080, USA., Dever DP; Graphite Bio, Inc., South San Francisco, CA 94080, USA., Porteus MH; Kamau Therapeutics, Inc., South San Francisco, CA 94080, USA. Electronic address: mporteus@kamautx.com., Wienert B; Graphite Bio, Inc., South San Francisco, CA 94080, USA. Electronic address: b.wienert@outlook.com. |
| Source: | Molecular therapy : the journal of the American Society of Gene Therapy [Mol Ther] 2025 Apr 02; Vol. 33 (4), pp. 1407-1419. Date of Electronic Publication: 2025 Feb 28. |
| Publication Type: | Journal Article |
| Journal Info: | Publisher: Cell Press Country of Publication: United States NLM ID: 100890581 Publication Model: Print-Electronic Cited Medium: Internet ISSN: 1525-0024 (Electronic) Linking ISSN: 15250016 NLM ISO Abbreviation: Mol Ther Subsets: MEDLINE |
| Database: | MEDLINE Ultimate |
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